{"id":"W1569820390","doi":"10.1002/jgm.2768","title":"Adeno‐associated virus 8‐mediated gene therapy for choroideremia: preclinical studies in <i>in vitro</i> and <i>in vivo</i> models","year":2014,"lang":"en","type":"article","venue":"The Journal of Gene Medicine","topic":"Retinal Development and Disorders","field":"Biochemistry, Genetics and Molecular Biology","cited_by":31,"is_retracted":false,"has_abstract":true,"ca_institutions":"","funders":"Foundation Fighting Blindness; Pennsylvania Department of Health; University of Pennsylvania; Research to Prevent Blindness; F. M. Kirby Foundation; Choroideremia Research Foundation","keywords":"Choroideremia; In vivo; Adeno-associated virus; Biology; Genetic enhancement; In vitro; Retinal; Retinal pigment epithelium; Rab; Retinal degeneration; Gene delivery; Molecular biology; Western blot; Virology; Cell biology; Recombinant DNA; Gene; Vector (molecular biology); Biochemistry; Genetics","routes":{"ca_aff":false,"ca_fund":true,"ca_venue":false,"about_ca":false,"invisible_to_affiliation_only":true},"retraction":null,"screen":null,"direct_labels":[],"prediction":{"model_version":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.001274624,0.001232647,0.0006521468,0.0007639501,0.0003329605,0.0004334519,0.0006139179,0.0006313231,0.002530531],"category_scores_gemma":[0.0003766481,0.0002363996,0.0005426945,0.0002893884,0.0007059183,0.0005762063,0.0001806343,0.001460162,0.0007477261],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0007080089,"about_ca_system_score_gemma":0.0006732412,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.001305035,"about_ca_topic_score_gemma":0.001621182,"domain_scores_codex":[0.9995609,0.0001438621,0.00003960027,0.00007071688,0.00009993653,0.00008500808],"domain_scores_gemma":[0.9995512,0.00009842947,0.0001416809,0.00005788617,0.00004893848,0.0001018259],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"bench_or_experimental","study_design_gemma":"bench_or_experimental","study_design_scores_codex":[0.001285999,0.002136644,0.0002612439,0.0003085136,0.00004605081,0.0001261229,0.00008892039,0.0006732126,0.9858393,0.0004444023,0.0005029875,0.008286522],"study_design_scores_gemma":[0.0005331177,0.03458866,0.001971341,0.00005999233,0.000149958,0.0005264758,0.00004796861,0.001182454,0.95417,0.0001656203,0.006584095,0.00002031983],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9601832,0.01041055,0.01681033,0.0006147275,0.0002417379,0.00154364,0.001469994,0.0006362017,0.008089663],"genre_scores_gemma":[0.9713572,0.007528638,0.01264336,0.0001467166,0.00007979093,0.000908159,0.001535412,0.0001217307,0.00567901],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.002530531,"threshold_uncertainty_score":0.008465528,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.04159326182806776,"score_gpt":0.3213658012579557,"score_spread":0.2797725394298879,"validation_status":"score_only:v0-immature-baseline","note":"Baseline scores from an immature model (maturity gate not passed). Scores rank; they never assert a category."}}